KAYFANDA (odevixibat) - Cholestatic pruritus in Alagille syndrome

The Committee deems that the clinical benefit of KAYFANDA (odevixibat) is substantial in the MA indication.

Considering:

• the results of a randomised, placebo-controlled, phase 3 trial conducted in children with a rare disease, demonstrating a clinically relevant effect in terms of reducing pruritus (reduction in scratching severity score) in 52 patients;
• the safety profile of odevixibat;
• but also the absence of data on cholestasis complications and recourse to transplantation due to refractory pruritus,

the Committee deems that KAYFANDA (odevixibat) provides a moderate clinical added value (CAV III) in the current care pathway.